Core Insights - Pharming Group forecasts 2026 revenue between $405 million and $425 million, representing an 8% to 13% growth compared to 2025, exceeding analyst expectations of approximately $398 million [1][10] - The company emphasizes continued revenue growth, disciplined spending, and multiple clinical readouts expected over the next two years [4] Financial Guidance - For 2026, Pharming expects operating expenses to be between $300 million and $335 million, with a gross margin of approximately 90% [1][12] - The company reported preliminary 2025 revenue of around $376 million, a 27% increase from 2024, with Joenja revenue surpassing $50 million [7][9] Commercial Portfolio - RUCONEST is projected to generate $318 million in 2025, reflecting a 26% year-over-year growth, while Joenja is expected to reach $58 million, up 29% [9] - CEO Fabrice Chouraqui highlighted RUCONEST's durability and its role as a reliable treatment for hereditary angioedema patients, noting its complex manufacturing process [5][14] Regulatory and Pipeline Updates - Pharming plans to request a Type A meeting with the FDA in March regarding pediatric dosing for Joenja, which has been excluded from 2026 revenue guidance due to regulatory uncertainties [2][6][11] - Management anticipates two phase II readouts for leniolisib (Joenja) in H2 2026 and an EMA decision in H1 2026, alongside a pivotal trial for napazimone (KL1333) with results expected in late 2027 [3][16][21] Market Dynamics - RUCONEST's growth is expected to be driven by U.S. volume growth, with a 3% price increase implemented in line with CPI [14] - The company has not included potential revenue from reclassification of VUS patients in its guidance due to timing uncertainties [15]

Summary of Pharming Group 2026 Investor Day Company Overview - **Company**: Pharming Group (NasdaqGM:PHAR) - **Event**: 2026 Investor Day held on February 3, 2026 - **CEO**: Fabrice Chouraqui Financial Performance - **2025 Revenue**: Approximately $376 million, a 27% increase from 2024, slightly exceeding previous guidance [5][6] - **Reconnect Revenue**: $318 million, a growth of 26% year-over-year [7] - **Joenja Revenue**: $58 million, a growth of 29% [7] - **Gross Margin**: Expected around 88%-89% for 2025, excluding a $5 million milestone payment to Novartis [8] - **Cash Position**: $181 million in cash and marketable securities at the end of 2025 [9] 2026 Financial Guidance - **Expected Revenue**: Between $405-$425 million, implying growth of 8%-13% over 2025 [9] - **Reconnect Growth**: Mid-single-digit growth expected, with a decline in ex-U.S. revenues due to market exit [9] - **Joenja Growth**: Anticipated to accelerate, with annual growth around 10 percentage points higher than in 2025 [9] - **Operating Expenses**: Expected between $300 million-$335 million, including over $60 million in R&D investments [10][11] Pipeline and Product Development - **Leniolisib**: Focused on high-prevalence primary immunodeficiencies (PIDs) and is in pivotal phase 2 trials [4][31] - **KL1333 (napazimod)**: Targeting primary mitochondrial disease, also a billion-dollar opportunity [4] - **Joenja**: Committed to pediatric label expansion despite FDA response; Type A meeting scheduled for March [3][10] - **Market Potential**: Both Reconnect and Joenja have over $1 billion sales potential each [2] Market Dynamics - **Reconnect**: Positioned as a cornerstone treatment for difficult-to-treat patients, with a mechanism that provides reliability during crises [2][30] - **Joenja**: Growth opportunities identified in APDS, with potential to reclassify about 250 VUS patients as APDS over time [3] - **CVID Expansion**: Plans to expand into larger PIDs and CVID, unlocking a much larger market [4] Clinical Insights - **Leniolisib's Role**: Investigated for treating immune dysregulation in PIDs, with a strong scientific rationale and patient need [55][56] - **CVID and APDS**: Clinical overlap noted, with a significant portion of CVID patients exhibiting similar characteristics to APDS [44][46] - **Patient Experience**: Anecdotal evidence suggests that leniolisib has provided hope and improved quality of life for patients with CVID [53] Regulatory and Approval Status - **FDA Engagement**: Plans to engage with the FDA regarding pediatric approval for Joenja and address concerns raised in the recent CRL [15][29] - **EMA Approval**: Expected approval for leniolisib in the first half of 2026 [32] Conclusion - Pharming Group is positioned for substantial growth with a strong pipeline and financial performance. The company is focused on expanding its market presence in rare diseases, particularly through innovative treatments like leniolisib and Joenja, while maintaining financial discipline and strategic investments in R&D.

Core Insights - Pharming Group N.V. has announced its financial guidance for 2026, projecting total revenues between US$405 million and US$425 million, representing an 8% to 13% growth [3][7][8] - The company will highlight its advancing clinical-stage pipeline, focusing on two major programs targeting primary immunodeficiencies and mitochondrial diseases during its Investor Day [2][4][6] Financial Guidance - Total revenue guidance for 2026 is set between US$405 million and US$425 million, driven by growth in its commercial products [3][8] - Total operating expenses are expected to be between US$330 million and US$335 million, primarily due to increased R&D expenses related to ongoing clinical trials [7][8] Pipeline Overview - Leniolisib is being developed for primary immunodeficiencies with immune dysregulation, with ongoing Phase II trials expected to yield top-line data in the second half of 2026 [4][5] - Napazimone (KL1333) is being developed for mtDNA-driven mitochondrial disease, with a pivotal clinical trial ongoing and results anticipated in 2027 [6][9] Clinical Programs - Leniolisib is currently approved as Joenja in the U.S. and is the first targeted treatment for activated PI3Kδ syndrome, with potential applicability across broader patient populations [5][6] - Napazimone (KL1333) aims to become the first standard of care for mtDNA-driven mitochondrial disease, addressing significant unmet medical needs [6][9] Investor Day Details - The Investor Day will feature presentations from clinical experts discussing disease biology and the potential impact of Pharming's programs [10][16] - The event is scheduled for February 3, 2026, from 10:00 a.m. to 12:00 p.m. EST, and will be available via webcast [11]

Summary of Pharming Group Conference Call Company Overview - Pharming Group is a Dutch-based biotech company primarily operating in the US, known for its recombinant protein platform and the drug RUCONEST, which has been on the market for 10 years [3][4] - The company has transitioned from a one-asset firm to a high-growth biotech with two market assets, RUCONEST and Joenja, both experiencing double-digit growth [3][5] Financial Performance - Pharming reported strong third-quarter results, raising revenue guidance due to the growth of RUCONEST (29% year-on-year in Q4) and Joenja (35% growth) [5][6] - The company aims to maintain double-digit growth while investing in high-growth assets from generated cash [10][32] Product Insights RUCONEST - RUCONEST serves a specific segment of the Hemophilia A (HA) market, targeting patients who are not controlled by prophylactic treatments and have severe crises [7][9] - The drug's unique value proposition includes its ability to replace the missing protein and provide a fast onset of action through IV administration [7][8] - Pharming does not foresee any new treatments that could significantly impact RUCONEST's market share, expecting continued growth [9][10] - The company has exited non-US markets due to financial unsustainability, with less than 1.5% of RUCONEST sales coming from those regions [11][12] Joenja - Joenja is positioned as a high-growth drug with potential to become Pharming's first billion-dollar product, currently indicated for APDS (Activated PI3K Delta Syndrome) [14][15] - The company is expanding Joenja's indications to pediatric patients, with FDA approval expected by the end of January, which could increase the addressable market by 30% [16][18] - There is also an opportunity to reclassify Variant of Uncertain Significance (VUS) patients as APDS patients, potentially increasing the patient base by 50% [17][18] Market Expansion - Joenja has launched successfully in the UK, with plans for further expansion into Europe and Japan, pending regulatory approvals [23][24] - The company is focused on ensuring appropriate pricing that reflects the drug's value in new markets [24] Acquisition and Pipeline - Pharming's acquisition of Abliva for KL1333, aimed at treating primary mitochondrial disease, is seen as a strategic addition to its rare disease portfolio [25][26] - KL1333 has a well-defined patient population and is in registrational phase 2 trials, with significant market potential [26][28] Operational Strategy - The company is focused on optimal capital allocation to drive growth and has recently cut 20% of G&A headcount to reallocate resources towards growth catalysts [32][33] - Pharming emphasizes the importance of disciplined management to enhance value creation in the long term [33] Conclusion - Pharming Group is positioned for continued growth through its existing products and strategic acquisitions, with a strong focus on expanding its market presence and optimizing operational efficiencies [32][33]

Financial Data and Key Metrics Changes - Total revenues grew by 30% in Q3 2025 compared to the same quarter last year, reaching $97.3 million [2][19] - Operating profit increased to $15.8 million, nearly four times last year's result [2][19] - Operating cash flow was $32 million, restoring cash position to levels seen at the end of 2024 [2][19] - Gross profit grew by 33% to $90.2 million, resulting in a gross margin of 93% compared to 91% in the same quarter of 2024 [19][63] Business Line Data and Key Metrics Changes - Ruconest revenue increased by 29% year-on-year to $82 million, driven by new prescribers and patient enrollments [3][7] - Joenja's revenue grew by 35% year-on-year, generating $15.1 million, with a 25% increase in patients on treatment [3][11] - The company anticipates adding new growth sources for Joenja with pediatric indications and geographic expansion [3][12] Market Data and Key Metrics Changes - The company upgraded its full-year 2025 revenue guidance to $360-$365 million from the previous $335-$350 million [4][21] - The strong performance in Q3 reflects a solid foundation for growth, transitioning from a single-asset company to a biotech with two high-growth products [4][5] Company Strategy and Development Direction - The company aims to optimize capital deployment towards high-growth initiatives and enhance financial discipline [4][25] - Pharming is focused on building a leading global rare disease company, leveraging its strong capabilities in rare diseases [5][24] - The company is advancing its pipeline with two programs that have over $1 billion sales potential each [5][24] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the long-term growth prospects of Ruconest, emphasizing its unique value for severe HAE patients [9][24] - The anticipated pediatric launch of Joenja is expected to be a significant growth driver, with preparations on track for a January 2026 approval [12][24] - Management highlighted the importance of maintaining financial discipline and ensuring efficient capital allocation [25][21] Other Important Information - The company is taking steps to reduce G&A headcounts to optimize capital deployment [4] - The recent publication in Cell suggests a higher prevalence of APDS, potentially expanding the patient population significantly [15][16] Q&A Session Summary Question: Impact of new oral therapy on Ruconest and revenue from pediatric patients on Joenja - Management does not see Ruconest competing with sebetralstat, as it serves a different patient population with more severe needs [28][29] - For Joenja, a significant number of pediatric patients identified are expected to convert to therapy quickly after approval [29] Question: Timeline for Ruconest withdrawal outside the US and competitive threats - The withdrawal is planned for the first half of next year, driven by financial sustainability concerns [36] - Management believes Ruconest serves a unique patient population and does not anticipate significant competitive threats [37] Question: Revenue guidance uplift and contributions from Ruconest and Joenja - The revenue guidance uplift is attributed to strong performances from both Ruconest and Joenja, with expectations for continued growth [54][55] Question: Capital allocation priorities and M&A plans - The company is focused on disciplined capital allocation and is not in a rush for M&A, looking for value-accretive opportunities [44][45] Question: Progress on the basket PID trial - The phase two proof-of-concept study is progressing well, with expected readouts in the second half of 2026 [46]